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Lumacaftor

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Precision Medicine for Long QT Syndrome Type 2: Lumacaftor Corrects Trafficking Defects and Shortens QT Interval
Posted inCardiology news

Precision Medicine for Long QT Syndrome Type 2: Lumacaftor Corrects Trafficking Defects and Shortens QT Interval

Posted by By MedXY 09/28/2026
A phase 2 clinical study demonstrates that lumacaftor safely shortens QTc in LQT2 patients with KCNH2 trafficking defects, supported by patient-specific cardiomyocyte models validating the mechanism.
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